There was no difference in cognitive outcomes when African infants with post-infectious hydrocephalus, an accumulation of cerebrospinal fluid in the brain, were treated with a shunt or a two-part procedure designed to divert the flow of fluid and cauterize certain areas of the brain, a small randomized trial found.
Infants who underwent endoscopic third ventriculostomy with choroid plexus cauterization (ETV-CPC) showed no difference in median cognitive scores at 12 months compared to infants treated with ventriculoperitoneal shunting, reported Abhaya V. Kulkarni, MD, of the University of Toronto Hospital for Sick Children, and colleagues.
There was also no difference in language or motor skills or in brain volume between the two procedures as performed in Uganda, the authors wrote in the New England Journal of Medicine.
They said that there is “uncertainty” regarding the benefits of the two procedures for infants with this condition. While shunting is technically straightforward, with a lower rate of failure in the months after surgery, the authors said, it has a higher long-term failure rate that often requires urgent surgery. In addition, “shunt failure can be fatal in regions of the world where there is no access to immediate neurological care,” they wrote.
But shunting is also thought to reduce the ventricle size more than ETV-CPC, which could lead to a better cognitive outcome for the patient, though the authors added that “this difference between procedures has been shown inconsistently.”
“Others have suggested that shunts are a better treatment in regard to neurologic development or brain growth, but we found this was not the case,” co-author Benjamin Warf, MD, of Boston Children’s Hospital said in a statement. “There are many advantages to avoiding lifelong dependence on a shunt.”
Researchers randomized 100 Ugandan infants with post-infectious hydrocephalus, who were less than 180 days of age, with a mother who was over 18, and lived in an area where they could complete follow-up. They were evaluated via the cognitive scaled score on the Bayley Scales of Infant Development, Third Edition, 12 months following surgery.
Overall, 42 infants underwent endoscopic ventriculostomy and 48 underwent placement of a ventriculoperitoneal-shunt. Infants were a mean age of about 3 months, and there were around 40% girls in both groups. Head circumference and cerebrospinal fluid volume was greater in the shunt group, the author said, but noted no other significant between-group differences at baseline.
There was no significant difference in median cognitive scores between the two groups at 12 months (4 in ETV-CPC group versus 2 in shunt group, 95% CI -2 to 0, P=0.35). Kulkarni and colleagues also found no significant difference in expressive language score, receptive language score, general motor score or fine motor score.
While there was no difference in brain volume at 12 months between groups, median cerebrospinal fluid volume was significantly lower in the shunt group than in the ETV-CPC group.
There were 18 treatment failures in the ETV-CPC group compared to 12 in the shunt group, but the difference was non-significant. In a statement, Warf characterized that finding as surprising.
“ETV/CPC is expected to have a higher short term failure rate, whereas shunts tend to fail more over time,” Warf said. “We expect that there will be no further ETV/CPC failures, which nearly all occur within 6 months, but that the shunts will continue to fail as we follow this cohort.”
There were nine deaths, two of which were treatment-related. One patient in the ETV-CPC group had treatment failure 8 months after surgery, the authors said, and one patient started in the ETV-CPC group, crossed over to shunting and developed ventriculitis (inflammation of the ventricles) and subdural empyema (a collection of pus in certain brain matter).
But they noted that “the overall developmental outcome was relatively poor for both treatments,” with nearly all patients starting with abnormally low brain volume at baseline, and only a quarter having normal brain volume by year 1, they said.
Limitations to the data include that it only addressed postinfectious hydrocephalus, that the single center nature of the trial limits generalizability, and a relatively short follow-up period of 1 year.
“We will continue to follow these children, but the results further raise our level of confidence in recommending ETV/CPC as the initial treatment for most infants with hydrocephalus,” Warf said.
This study was supported by the NIH, the National Institute of Child and Human Development, and the Fogarty International Center.
Kulkarni disclosed no conflicts of interest.
One co-author disclosed support from General Electric Global Research.
F. Perry Wilson, MD, MSCE Assistant Professor, Section of Nephrology, Yale School of Medicine and Dorothy Caputo, MA, BSN, RN, Nurse Planner